MX2018011114A - Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. - Google Patents

Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.

Info

Publication number
MX2018011114A
MX2018011114A MX2018011114A MX2018011114A MX2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A
Authority
MX
Mexico
Prior art keywords
crispr
cas
compositions
related methods
treating beta
Prior art date
Application number
MX2018011114A
Other languages
English (en)
Spanish (es)
Inventor
Leah Gori Jennifer
A Barrera Luis
Original Assignee
Editas Medicine Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2018011114A publication Critical patent/MX2018011114A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0641—Erythrocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MX2018011114A 2016-03-14 2017-03-14 Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. MX2018011114A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662308190P 2016-03-14 2016-03-14
US201762456615P 2017-02-08 2017-02-08
PCT/US2017/022377 WO2017160890A1 (en) 2016-03-14 2017-03-14 Crispr/cas-related methods and compositions for treating beta hemoglobinopathies

Publications (1)

Publication Number Publication Date
MX2018011114A true MX2018011114A (es) 2019-02-20

Family

ID=58413206

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018011114A MX2018011114A (es) 2016-03-14 2017-03-14 Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.
MX2024012899A MX2024012899A (es) 2016-03-14 2018-09-13 Metodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatias

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024012899A MX2024012899A (es) 2016-03-14 2018-09-13 Metodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatias

Country Status (11)

Country Link
US (2) US20200255857A1 (enExample)
EP (1) EP3430142A1 (enExample)
JP (2) JP2019508051A (enExample)
KR (2) KR102723347B1 (enExample)
CN (4) CN117802102A (enExample)
AU (2) AU2017235333B2 (enExample)
CA (1) CA3017956A1 (enExample)
IL (2) IL313038A (enExample)
MX (2) MX2018011114A (enExample)
SG (1) SG11201807859WA (enExample)
WO (1) WO2017160890A1 (enExample)

Families Citing this family (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
EP3262173A2 (en) 2015-02-23 2018-01-03 Crispr Therapeutics AG Materials and methods for treatment of human genetic diseases including hemoglobinopathies
SG11201706766WA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
EP3371306B8 (en) 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
HRP20240842T1 (hr) * 2016-11-02 2024-10-11 Universität Basel Imunološki uočljive varijante stanične površine za upotrebu u staničnoj terapiji
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018200597A1 (en) * 2017-04-24 2018-11-01 Seattle Children's Hospital (dba Seattle Children's Research Institute) Homology directed repair compositions for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3645721A1 (en) * 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592C1 (en) * 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
US20210180091A1 (en) * 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN111556893A (zh) * 2017-11-06 2020-08-18 爱迪塔斯医药股份有限公司 免疫疗法t细胞中cblb的crispr-cas9编辑的方法、组合物和组分
MX2020005849A (es) 2017-12-05 2020-11-06 Vertex Pharma Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.
AU2018383712B2 (en) * 2017-12-11 2025-09-04 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3762496A2 (en) * 2018-03-07 2021-01-13 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
AU2019234922A1 (en) * 2018-03-14 2020-10-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US20200149070A1 (en) * 2018-04-24 2020-05-14 Ligandal, Inc. Methods and compositions for genome editing
CA3100019A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US20220033856A1 (en) * 2018-09-11 2022-02-03 Université de Paris Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3887521A1 (en) * 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3891276A4 (en) * 2018-12-05 2022-07-27 Fred Hutchinson Cancer Center PRODUCTION OF GENETICALLY MODIFIED CELLS WITH REDUCED AND MINIMAL MANIPULATION
CN111321171A (zh) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN112011576A (zh) * 2019-05-31 2020-12-01 华东师范大学 Crispr基因编辑技术在治疗地中海贫血中的应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN112979823B (zh) * 2019-12-18 2022-04-08 华东师范大学 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
CN111876416B (zh) * 2020-07-01 2021-09-03 广州瑞风生物科技有限公司 激活γ-珠蛋白基因表达的方法和组合物
AU2022381552A1 (en) * 2021-11-02 2024-05-09 Incisive Genetics, Inc. Compositions and methods for preventing, ameliorating, or treating sickle cell disease
CN114848851A (zh) * 2022-04-29 2022-08-05 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） 治疗β-地中海贫血的药物
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2025038164A1 (en) * 2023-08-15 2025-02-20 University Of Massachusetts Nick resection in cancer
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
RU2018109507A (ru) * 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
SG11201500852WA (en) * 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
AU2013352156B2 (en) * 2012-11-27 2018-12-06 Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
EP4435110A3 (en) * 2013-06-05 2025-01-01 Duke University Rna-guided gene editing and gene regulation
CN111218447B (zh) * 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11242525B2 (en) * 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
SG11201706766WA (en) * 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法

Also Published As

Publication number Publication date
KR20180120752A (ko) 2018-11-06
CN118127006A (zh) 2024-06-04
US20240360471A1 (en) 2024-10-31
CA3017956A1 (en) 2017-09-21
MX2024012899A (es) 2024-11-08
IL261714A (en) 2018-10-31
KR102532663B1 (ko) 2023-05-16
KR102723347B1 (ko) 2024-10-28
WO2017160890A1 (en) 2017-09-21
US20200255857A1 (en) 2020-08-13
CN117802102A (zh) 2024-04-02
IL313038A (en) 2024-07-01
IL261714B2 (en) 2024-10-01
SG11201807859WA (en) 2018-10-30
IL261714B1 (en) 2024-06-01
AU2017235333A1 (en) 2018-10-04
KR20230070331A (ko) 2023-05-22
AU2023214243A1 (en) 2023-08-31
JP2019508051A (ja) 2019-03-28
JP2023075166A (ja) 2023-05-30
CN109153994A (zh) 2019-01-04
AU2017235333B2 (en) 2023-08-24
EP3430142A1 (en) 2019-01-23
CN117821458A (zh) 2024-04-05

Similar Documents

Publication Publication Date Title
MX2018011114A (es) Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.
IL268406A (en) Preparations and methods for treating hemoglobin diseases
ZA202006627B (en) Methods and compositions for treating cancer
ZA201903327B (en) Compositions and methods for the induction of cd8+ t¿cells
IL264034A (en) Crispr/cas9-based compositions and methods for treating cancer
IL260257A (en) Preparations and methods for the treatment of hemoglobinopathies
IL276808A (en) Preparations and methods for the treatment of cancer
MX2019003623A (es) Composiciones y metodos para el tratamiento de afecciones oftalmicas.
EP3556858A3 (en) Crispr/cas-related methods and compositions for treating cystic fibrosis
GB2541571A (en) Pharmaceutical compositions
MX2018009325A (es) Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
ZA201900960B (en) Methods and compositions for the treatment of cancer
SG11202103033WA (en) Compositions and methods for the treatment of presbyopia
MX2022015753A (es) Metodos y composiciones para tratamiento de epidermolisis ampollosa.
PH12018501279A1 (en) Compositions comprising 15-hepe and methods of using the same
IL268163A (en) Methods, compositions, and kits for treatment of cancer
SG11202012435UA (en) Compositions and methods for treating cancer
SG11202003539VA (en) Compositions and methods for treating liberibacter diseases and other bacterial diseases
ZA201807438B (en) Methods of treating ocular conditions
IL278978A (en) Preparations and methods for treating eczema
MX2022005888A (es) Metodos para tratar afecciones oculares.
MX2020012990A (es) Formulaciones de tegavivint y compuestos relacionados.
IL276203A (en) Compounds and methods for treating cancer
SG10201805890QA (en) Compositions and methods for treatment of abnormal cell growth
ZA201808232B (en) Compositions and methods for the treatment of cancer